Our overall goal is to develop a better understanding of the processes that occur during neurodegenerative disorders in order to facilitate therapies that may ultimately prolong cognitive or motor function in diseases such as Alzheimer’s (AD) and Parkinson’s diseases (PD), frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). A large part of our work involves the generation of constitutive and inducible model systems for the study of proteins that are thought to play a major role in disease progression including TDP-43, tau and lrrk2. These models comprise a critical resource for the CTRND as it strives to translate laboratory findings into diagnostics or therapies for human diseases. In particular we focus on cellular pathways altered in these mouse models that may play a contributory role in disease and study the formation of pathological species of protein that are hallmarks of the human condition. Understanding these pathways is key to developing therapies against these pathological proteins or the damage that they cause. In addition to our generation and characterization of novel model systems for human neurodegenerative diseases, we interact with other academic or pharmaceutical colleagues to determine the efficiency of novel therapies for the human diseases that we study. Recent and ongoing funding from the NIH (NINDS, NIA), DoD, ALSA, Alzheimer’s Association, and MJFF as well as private benefactors have been critical in helping our studies move toward the clinic.

Ongoing projects in her laboratory include:

Testing novel siRNA therapeutics against alpha-synuclein, a protein that is involved in PD

Generation of models of FTD and ALS to understand how changes to TDP-43 and/or progranulin cause human disease.

Utilizing our novel models for FTD and AD to understand how the tau protein causes disease and to identify ways through which the brain can be repaired.

Investigating the link between different pathological proteins involved in Recent Publications.